Study of Cavosonstat (N91115) in Patients With CF Homozygous for the F508del-CFTR Mutation (SNO-6)

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This will be a double-blind, randomized, placebo-controlled, parallel group study. The purpose of this study is to investigate the efficacy and safety of Cavosonstat (N91115) in adult patients with CF who are homozygous for the F508del-CFTR mutation and being treated with lumacaftor/ivacaftor (Orkambi™).

Condition or disease

Intervention/treatment

Phase

Cystic Fibrosis

Drug: CavosonstatDrug: Placebo

Phase 2

Detailed Description:

Primary Objective:

Assess the efficacy of N91115 at 12 weeks when added to preexisting treatment with lumacaftor/ivacaftor in adult patients with CF who are homozygous for the F508del-CFTR mutation

Secondary Objectives:

Assess the effect of N91115 added to lumacaftor/ivacaftor on safety

Assess the effect of lumacaftor/ivacaftor added to N91115 on the pharmacokinetics of N91115, lumacaftor, and ivacaftor

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of N91115 to Evaluate Efficacy and Safety in Patients With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation Treated With Lumacaftor/Ivacaftor

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Layout table for eligibility information

Ages Eligible for Study:

18 Years and older (Adult, Older Adult)

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

No

Criteria

Inclusion Criteria:

Patients must have been treated with lumacaftor/ivacaftor for at least 8 weeks prior to Day 1 (start of dosing)

A history of Sweat Chloride (SC) ≥ 60 mEq/L by quantitative pilocarpine iontophoresis test (QPIT) (either before or after starting lumacaftor/ivacaftor treatment)

Body weight ≥ 40 kg

ppFEV1 40 - 85 % predicted (inclusive) at screening

Oxygen saturation ≥ 90% breathing ambient air at screening

Exclusion Criteria:

Any acute infection that requires treatment or hospitalization within 2 weeks of Study Day 1

Colonization with organisms associated with more rapid decline in pulmonary status, such as Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus

Any change in the regimen for chronic therapies for CF lung disease (e.g., Pulmozyme®, hypertonic saline, Azithromycin, TOBI®, Cayston®) within 4 weeks of Study Day 1

Are pregnant, planning a pregnancy, or breast-feeding at screening

Blood hemoglobin < 10 g/dL at screening

Serum albumin < 2.5 g/dL at screening

Abnormal liver function defined as ≥ 3 x upper limit of normal (ULN)

History of abnormal renal function within 3 months of screening

History of ventricular tachycardia or other clinically significant ventricular arrhythmias